Using Patient Reported Outcomes to Inform Health Care Research and Practice

Abstract

Use of patient-reported outcomes (PROs) in addition to clinical (e.g., biomarkers, vitals, etc.) and organizational (e.g., admission, cost, etc.) outcomes are critical for integrating the patient perspective into medical product development, treatment algorithms, evaluation of care quality, measures of health-related quality of life, and more. Key regulatory bodies including Centers for Medicare and Medicaid Services, Patient-Centered Outcomes Research Institute, National Institute of Health Pragmatic Trial Collaboratory, Agency for Healthcare Research and Quality, and The United States Food and Drug Administration have urged for incorporation of PROs, typically in the form of PRO measures (PROMs) into clinical care and research trials as a means to increase patients’ perspectives into treatment development and delivery. While production and use of PROs and PROMs in research trials and clinical practice has exponentially increased, there remains a significant lack of knowledge of PRO and PROM adoption, administration, and interpretation to inform endpoints for research and clinical care. Therefore, this dissertation seeks to address the evidence gap by exploring various applications of health measures across different patient populations and settings to contribute information that will ultimately foster patient-centered, measurement-based care. The three components of this dissertation are outlined below to describe the range of patient populations, health contexts, and study designs used to provide doctoral level training in implementation strategies for health measurement and health services research. In the first project, we sought to determine which Patient Reported Outcome Measurement Information System (PROMIS) measures are associated with high-impact chronic pain (HICP) in patients status post shoulder arthroplasty (TSA) and propose cutoff scores to help guide future care recommendations. Identification of complex pain conditions, particularly HICP, is of growing interest among patients receiving total joint arthroplasty to better tailor pain interventions, as well as improve postoperative outcomes and quality of life. We performed a secondary analysis of a cohort of patients (n=227) who received a TSA at a single, academic medical center. Generalized linear models (GLM) estimated HICP from each PROMIS measure of physical function, pain interference, sleep disturbance, anxiety, and dyspnea individually, then in a combined model. Area under the curve (AUC) was calculated using receiver operator characteristic curves to assess accuracy of each PROMIS measure to estimate HICP status for patients receiving TSA. Bivariate GLM models and mean difference analyses revealed individuals with HICP had worse PROMIS scores in every included domain (all p-values <0.01). Only pain interference (AUC= 0.964) and physical function (AUC= 0.907) PROMIS measures met criteria (AUC>0.850) to accurately predict HICP. Cross validation and sensitivity analyses were performed to assess for differences among sociodemographic and surgical factors, and indicated little change in GLM models and cutoff scores for PROMIS measures. Further application and evaluation of these cutoff scores can be used to assist in refining assessment of outcomes for patients receiving TSA in registries, research trials, and clinical care. In the second project, we sought to perform a systematic review to understand what measures have been used to assess health-related quality of life (HRQoL) in patients experiencing acute bacterial skin and skin structure infection (ABSSSI) to guide measurement selection. ABSSSI can diminish patients’ HRQoL. The goal of this project was to identify and describe how HRQoL has been assessed in patients with ABSSSI in the literature to inform measurement selection for clinical antibacterial trials. We searched MEDLINE (PubMed), Embase (Elsevier), and CINAHL (EBSCO) for articles published between database inception and an updated search on October 14, 2022. We identified 37 articles that employed 42 distinct measures: 26 generic measures assessing HRQoL across multiple or singular domains, 14 condition-specific measures including repurposed dermatologic HRQoL metrics or measures relevant to surgical sites, and 2 author-developed questionnaires assessing treatment-related HRQoL. All findings were mapped to the Wilson and Cleary Model of HRQoL to explore domains of HRQoL captured in the evidence. We found reduced HRQoL for patients experiencing ABSSSI, which usually exhibited rapid onset and resolution related to symptom intensity. Pain had the greatest negative impact on HRQoL, and the most common HRQoL domain impacted was physical functioning. Progress has been made yet we did not identify a repurposed or novel tool that best captures HRQoL in patients with ABSSSI. For the final project of this dissertation, we sought to compare and contrast the health care experience for patients experiencing complicated urinary tract infections (cUTI) and their associated treating providers to inform future research on integration of patient reported outcome measures into patient-provider communication. Complicated urinary tract infections result in over 626,000 hospitalizations annually in the United States, yet little is known about the care experience of patients with cUTI and interactions with medical providers during care episodes. The perspectives of both patients receiving care for their cUTI and the providers navigating this complex condition are critical for informing how to increase patient perspective in research and clinical care. We used qualitative analysis to describe the care experience from patients who experienced a cUTI and care delivery from the perspective of their associated providers, and examine findings for similarities and differences. Single, semi-structured, individual, qualitative interviews were conducted via telephone or Zoom with all included patients and providers. We interviewed 12 patients and 16 providers, comprising 8 patient-provider dyads dyad (1 patient and corresponding (1) clinician) and 4 patient-provider triads (1 patient and corresponding (2) clinicians). Patient interviews averaged 44 (range 20–74) minutes and clinician interviews 21 (range 11–31) minutes. We used both structural and content analysis within and across interviews with patients and their treating providers, and organized findings using rapid, matrix-based techniques. Four concepts were identified: 1) time spent, 2) unknowns of care, 3) care coordination, and 4) discharge optimization. We found that increased or frequent interactions between patients and providers improved the care experience; however, a shared understanding of prognosis or cause for cUTI was critical for building patient-provider relationships. Additionally, there is a need for tools and strategies that promote care coordination across providers and settings, as well as a need to augment discharge preparedness and follow-up care. More research is needed to understand how PRO and/or PROMs can help to address concepts identified in this analysis.